AQVESME is the only FDA-approved medicine for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or ...

An oral pyruvate kinase (PK) activator, mitapivat becomes the only approved option for treating anemia in both non-transfusion- and transfusion-dependent patients with alpha- or beta-thalassemia, ...

FDA approves Agios' Aqvesme for anemia in alpha and beta thalassemia, backed by Phase 3 data and a REMS safety program.

Vanda also sells sleep disorder drug Hetlioz, which reached the market in 2014, and relapsing multiple sclerosis treatment Ponvory, which was approved in 2021. The FDA is scheduled on Jan. 7 to decide ...

The US FDA has approved Mitapivat (Aqvesme), the first oral therapy for anemia in adults with alpha or beta thalassemia, ...

Approval was based on the phase 3 ENERGIZE and ENERGIZE-T studies evaluating the safety and efficacy of mitapivat in adults with alpha- or beta-thalassemia.

Credit: Shutterstock. Zynteglo is a one-time gene therapy that works by adding functional copies of a modified form of the β-globin gene into a patient’s own hematopoietic stem cells. The Food and ...

The US Food and Drug Administrations (FDA) approval of the first-ever oral pill for anaemia in adults with thalassemia marks a historic milestone and can be a game changer in the management of the ...

DelveInsight’s 'Beta Thalassemia Pipeline Insight – 2023' report provides comprehensive global coverage of available, marketed, and pipeline beta thalassemia therapies in various stages of clinical ...

The month of May saw major announcements in breakthrough therapies by companies working on the Beta-Thalassemia curatives - LentiGlobin BB305 and Luspatercept. The month of May saw major announcements ...

In 1976, my final year of medical school, I travelled abroad and spent several months working in the hematology clinic at Hadassah Hospital, in Jerusalem. Every day, I attended to children and ...